Tests & Treatments – State of Healthhttps://ww2.kqed.org/stateofhealth
KQED Public Media for Northern CASun, 18 Mar 2018 16:58:56 +0000en-UShourly1https://wordpress.org/?v=4.2.290498487Health Care Revamp At The L.A. County Jailshttps://ww2.kqed.org/stateofhealth/2018/02/27/health-care-revamp-at-the-l-a-county-jails/
https://ww2.kqed.org/stateofhealth/2018/02/27/health-care-revamp-at-the-l-a-county-jails/#respondTue, 27 Feb 2018 23:21:44 +0000https://ww2.kqed.org/stateofhealth/?p=362806Michael Callahan, an outgoing 43-year-old carpenter, landed in a Los Angeles County jail last September because of what he said were “bad decisions and selling drugs.”

He had uncontrolled diabetes and high blood pressure when he arrived, but his health was the last thing on his mind. Consumed by a meth addiction, he hadn’t taken his medications for months. “When I got here, I was a wreck,” said Callahan, who is stocky and covered in tattoos. “My legs were so swollen that if I bumped them they would break open.”

By January, however, his diabetes was improving and his blood pressure had dropped. Now, he takes his medications daily and sees a doctor every two months. Even as he counts the days until his release this summer, Callahan knows he is getting much-needed medical care. “I’m where I need to be, not where I want to be,” he said.

Callahan’s situation is counterintuitive: He may end up leaving jail healthier than when he arrived. Officials at the Los Angeles County Department of Health Services hope to see more cases like his as they embark on an ambitious effort to improve health care for jail inmates. Their project follows decades of complaints, lawsuits and reports of poor medical and mental health care at the Los Angeles County jails, which house about 18,000 inmates on any given day.

The county’s overhaul is designed to raise the quality of health care behind bars and better equip inmates to manage their health after they are released. But the challenges are enormous – the population is disproportionately sick, and the jails weren’t designed to be medical facilities.

The innovative effort at one of the nation’s biggest jail systems is based on a logical premise: Inmates don’t stay in jail for long – the average stay is just 60 days – so it’s a crucial opportunity to diagnose and begin treating their diseases.

“People are there for just a blip in time, days, weeks, months … and they’re returning back to the community,” said Mark Ghaly, director of community health for the county Department of Health Services. “What happens in the jail matters.”

The county health agency took over medical care in the jails from the LA County Sheriff’s Department in 2015 and started revamping the system in earnest last year.

The health clinic at a Los Angeles County jail on January 17, 2018. Inmates wait on a bench to get medications or see medical providers. (Heidi de Marco/KHN)

The main health clinic at the Men’s Central Jail in downtown Los Angeles is located just inside a large metal gate. Inmates there and at all the LA county jails can get a wide variety of medical and behavioral care. “It’s a giant health system and it’s complex,” said Margarita Pereyda, chief medical officer of correctional health services for LA County. “We are a hybrid between a hospital and an ER and an outpatient kind of environment.”

Part of the plan is to make clinics inside the jails more like ones on the outside. That means assigning inmates to primary care doctors to manage their chronic diseases and getting them appointments and medications quickly. It also means expanding treatment for mental health and substance abuse and referring those who need advanced medical or behavioral care to specialists who work for the county.

It’s a monumental job: Nearly half of all inmates have at least one chronic disease, including about 450 who have HIV and 900 with diabetes. About two-thirds of inmates are addicted to drugs or alcohol, and about a quarter have serious mental illnesses.

“Very few people have chronic illness under good control,” Ghaly said. ”The jails have largely become treatment facilities.”

To improve inmates’ access to care, county officials launched a physician recruitment effort this month. They released a series of online videos featuring medical providers with the slogan “Mission Possible.” As an incentive, they are offering to pay up to $120,000 in medical school debt for each of the new hires who need it. That strategy has been used to lure doctors to low-income communities around the United States.

Esther Lim, who directs the jails project at the American Civil Liberties Union of Southern California, said she is optimistic care will improve, but she still hears daily from inmates about delays in appointments and medications. And, she said, people are still dying inside the LA County jails – an average of 25 each year, according to the health department.

“It’s an indication that there is something wrong, that the delivery of medical care is still poor,” Lim said. Overcrowding can result in inmates’ health being neglected and deteriorating over time, she said. County health officials acknowledge the situation is not going to change overnight. The county is “making some great headway,” but “there are some things that you can change more quickly than others,” said Ed Matzen, clinical nursing director for the jails.

Public health nurse Martha Tadesse speaks with a patient at a Los Angeles County jail on January 17, 2018. (Heidi de Marco/KHN)

Lello Tesema, a primary care physician and director of population health for the county jails, said many of her patients have gone without care on the outside for a long time. As soon as she gets a new patient, Tesema takes a medical and personal history. Then she creates a plan with the patient, knowing she only has a limited amount of time to implement it.

One early morning in January, Tesema examined Callahan, the carpenter, on an exam table in a room just off a busy corridor around the corner from his dorm-style cell. She said the swelling in his legs had diminished and his blood sugar level was looking good. “We’re moving in the right direction,” she told him. On the way back to his bunk, Callahan stopped at a window to pick up a pill for his diabetes.

Tesema said she worries about the health of her patients after they get out of jail even though they leave with a referral to a county clinic and 30 days’ worth of medication – up from three days in the past. “Often I see patients come back and a lot of the successes that happened while they were here end up diminishing after they leave,” she said.

Tesema and other medical providers in the jail must manage the inherent tension between safety and medical care. Sometimes, doctors have to see patients in their cells or treat them when they are handcuffed, Tesema said.

Jason Wolak, a captain in the medical services bureau of the Sheriff’s Department, said deputies are making an effort to get more inmates to medical appointments. “We’re the Uber for medical,” he said. He added that the Sheriff’s Department needs more staff, especially for transporting inmates to outside specialists or to the county-run hospitals.

Since patients also are going to court, attending classes or meeting with their lawyers, scheduling medical visits can be a challenge, Ghaly said. “There’s a high no-show rate to appointments.”

Pereyda said the new system for providing care at the jails depends on current doctors changing their mindset – things as simple as calling people patients rather than inmates.

“We can figure out the logistics and we can figure out the resources, but shifting the way people think and act is going to be our biggest challenge,” she said. Hiring new doctors who believe in the mission of health care behind bars will help, she added.

Among some inmates, attitudes about their own health are already beginning to shift.

Callahan said he’s determined to stay sober and continue monitoring his health when he gets out. “I’m 43 years old and that’s not the age to be screwing around with diabetes,” he said.

]]>https://ww2.kqed.org/stateofhealth/2018/02/27/health-care-revamp-at-the-l-a-county-jails/feed/0362806There’s a Cure for Hepatitis C. Why Are So Many People Still Dying from It?https://ww2.kqed.org/stateofhealth/2017/11/30/theres-a-cure-for-hepatitis-c-why-are-so-many-people-still-dying-from-it/
https://ww2.kqed.org/stateofhealth/2017/11/30/theres-a-cure-for-hepatitis-c-why-are-so-many-people-still-dying-from-it/#commentsThu, 30 Nov 2017 13:00:24 +0000https://ww2.kqed.org/stateofhealth/?p=362025A small purple-and-white sign hangs on the waiting room wall in Highland Hospital’s emergency department in Oakland.

In English and Spanish it reads: “Our ER’s policy for patients 18 to 75 years old: HIV and Hepatitis C tests are done once a year if you are having other blood tests.”

It’s a policy that emergency room physician Doug White and his colleagues instituted four years ago. Since up to half of people with hepatitis C do not know they are infected, testing is the first step toward eradicating the deadly, blood-borne virus, he said.

“Hepatitis C is the leading cause of liver failure in the U.S.,” White said. “It’s the number one cause of liver cancer. Number one cause of liver transplants. It’s a phenomenon of epic proportion.”

Between 2.7 million and 3.9 million Americans have hepatitis C, according to the Centers for Disease Control and Prevention (CDC). The virus can remain dormant for years, and by the time symptoms arise, the organs may already be damaged. Except for flu, hepatitis C takes more lives than all other CDC-tracked infectious diseases combined — and that includes HIV, tuberculosis and other more prominent diseases.In 2015,19,629 Americans died from hepatitis C, mostly from liver disease caused by the virus.

For years the few treatments for hepatitis C had severe side effects and were not very effective at eliminating the virus from the body. But a new generation of powerful drugs came out in late 2013. These drugs have minimal side effects and usually require taking just one pill a day for two to three months. The cure rate is more than 90 percent.

Dr. White hopes Highland Hospital can be a leader in the fight, and a model for other hospitals tackling the hepatitis C epidemic.

“Hepatitis C has gone from a disease that I had no incentive to look for to diagnose, because I couldn’t do anything about it, to essentially a curable disease with treatment that’s well tolerated,” White said.

Highland staffers test nearly everyone who comes to the ER and gets blood drawn. They’ve built a robust electronic medical system that reminds medical staff to test for hepatitis C when doing other blood tests, as long as the patient hasn’t had this test in the past year. Highland was one of the first ERs to test for hepatitis C like this nationwide. That helps flag infections among low-income or undocumented patients who use the ER for primary care.

Dr. Doug White points to tubes used for hepatitis C blood tests. Highland Hospital’s emergency room has routinized their system so nearly every patient already getting blood drawn will also be tested for hepatitis C. (Laura Klivans/KQED)

And the work doesn’t end in the ER. If patients test positive, they get a call or visit from Mae Petti.

Petti has a desk a few floors above the emergency room. She spends hours making phone call after phone call. In her friendly, polite but insistent manner, she persists until she speaks with every patient on her list. As Highland’s hepatitis C linkage coordinator, Petti will contact patients again and again until they come in to get follow-up care.

Few hospitals have someone like Petti — her position at Highland is grant-funded. And her work is paying off: Highland has doubled the number of people who return for follow-up appointments. Now, patients can start getting treated for hepatitis C in as soon as two weeks. Previously, it took six months after a positive test before a patient began treatment.

San Francisco’s Mission to Stamp out the Virus

Across the bay, San Francisco has its own approach to the problem. A group there has been addressing hepatitis C since former Mayor Gavin Newsom assigned a specific task force to the issue in 2009. The group’s mission was first to recommend how the city should address hepatitis C. These days, they believe the threat of hepatitis C can be stamped out, and they think San Francisco is the place to do it.

At a recent task force gathering at San Francisco’s Department of Public Health, doctors, public health workers and many former hepatitis C patients — now cured — took their seats around a table.

After a dinner of pizza and salad, San Francisco’s viral hepatitis coordinator, Katie Burk, launched into a presentation. She listed reasons why she thinks San Francisco is a great candidate for elimination. First, she said, the city of San Francisco is small.

“That matters when you’re trying to grab folks from all parts of the city, and make sure they’re getting the care they need,” Burk said.

And there’s a historical commitment to fighting disease here.

“We have this really incredible HIV program infrastructure in San Francisco,” she said. “We’ve built a lot of what we’ve done with hepatitis C sort of on the back of that program, and learned from a lot of our successes and challenges.”

Hepatitis C is transmitted by blood — and these days most of that happens through shared needles. Stopping the spread of the virus is therefore inextricably connected with efforts to end addiction to injectable drugs. San Francisco has places to exchange dirty syringes for clean ones, and plenty of programs and clinics offer “medication-assisted treatment” — usually using buprenorphine, which helps people addicted to heroin and other opioids kick the habit.

San Francisco has another weapon too: a coalition specifically devoted to hepatitis C elimination, called End Hep C SF. It launched in 2016.

The End Hep C SF coalition has created something that experts consider crucial for the elimination of hepatitis C: an accurate estimate of how many residents harbor the virus, whether they are aware of it or not. In San Francisco, the current estimate is 12,000 people.

It’s unusual for a city to have a number like that to work with, because typically public health departments know only the number of people who have recently tested positive for hepatitis C. But San Francisco officials have gone farther: subtracting estimates of people who have been cured, died or moved away. They have calculated how many people probably have the virus, but have yet to be diagnosed.

Having that estimate is exciting, according to public health consultant Shelley Facente, who was also at the meeting, because it will allow the city to set goals of how many people to treat each year, and can offer an idea of when hepatitis C can largely be wiped out. The data have also helped identify target groups that have disproportionately high rates of hepatitis C in San Francisco, like transgender women.

Facente said roughly 4,500 San Franciscans already have been cured, using the new class of treatments for hepatitis C.

“That means this is doable,” Facente said, referring to elimination. “We can really make this happen. It’s very exciting that we’ve already made such progress. And now that we have even more information about what we need to do and where we need to go, it’s even going to get better from here.”

Remaining Barriers to Elimination

Back at Highland Hospital, patient June Bullock had an appointment to pick up a refill of pills for his hepatitis C treatment. He met with physician assistant Amy Smith, who guides Highland patients through the treatment. Smith handed Bullock his last pack of pills and asked if he had any questions. His wife, Alfreda, was the one who spoke up.

June (left) and Alfreda Bullock at Highland Hospital to pick up June’s final pack of medication for hepatitis C. (Laura Klivans/KQED)

“My daughter says nobody can drink behind him out of a straw,” Alfreda Bullock said.

“No,” Smith replied. “That’s not true.”

“There’s a whole bunch of stuff going on, you know what I’m saying?” Bullock said, explaining she’d heard a lot of misinformation about the virus.

Smith took the blame. “Yeah. Well, I’m sorry we haven’t covered that.” Smith then reviewed for them how hepatitis C is spread.

Research shows that to really curb hepatitis C, it takes a program like Highland’s: test widely, and then treat aggressively.

But, new hepatitis C infections still increased in California by 5.5 percent between 2011 and 2015, according to the state Department of Public Health.

That’s frustrating for people like Smith, who has seen many people get cured.

“There are a lot of good people out there doing good work,” she said. “I just don’t think all the pieces are in place just yet.”

Unaffordable drugs, for instance, are still one of the biggest barriers to treatment, Smith said.

The cheapest available drug retails at about $26,000 for a course that ranges from two to four months. Others can cost as much as $133,400. While insurance companies often negotiate lower prices, some have still tried to control costs by instituting limits on who qualifies for treatment.

Another problem is the nationwide opioid epidemic, which includes heroin abuse, often via needle. Many experts say that’s why hepatitis C infections are rising among people under 30 — although hepatitis C has traditionally been considered a disease of the baby boomers. This older generation grew up before the virus was identified, let alone subject to screening in the blood supply.

Some advocates say the fight against hepatitis C is especially difficult because society places little value on the lives of the people who tend to get the virus — current or former drug users, jail inmates and former prisoners.

Despite the challenges, efforts in the Bay Area show progress is still possible. The hope is that if a city like San Francisco can eliminate hepatitis C, others will follow suit.

]]>https://ww2.kqed.org/stateofhealth/2017/11/30/theres-a-cure-for-hepatitis-c-why-are-so-many-people-still-dying-from-it/feed/1362025Recreational or Procreational? Study Links Marijuana Use with More Frequent Sexhttps://ww2.kqed.org/stateofhealth/2017/10/27/recreational-or-procreational-study-links-marijuana-use-with-more-frequent-sex/
https://ww2.kqed.org/stateofhealth/2017/10/27/recreational-or-procreational-study-links-marijuana-use-with-more-frequent-sex/#commentsFri, 27 Oct 2017 19:37:12 +0000https://ww2.kqed.org/stateofhealth/?p=361766People who smoke marijuana reported having more sexual intercourse than non-users, according to a new study conducted by urologists at the Stanford University School of Medicine.

The results were modest but statistically significant: Non-users said they had engaged in sexual intercourse between five and six times in the previous month. Daily pot smokers reported having intercourse around seven times over that same period. The frequency was in-between for people who smoked marijuana less often, on a weekly or monthly basis: they reported having sex more than abstainers, but less than daily users.

“I was surprised,” said Dr. Michael Eisenberg, the study’s senior author and an assistant professor of urology at Stanford University Medical Center.

“The daily users for example, compared to the never users, reported about 20 more sexual encounters a year. So I think that is a significant difference,” he said.

It’s the first study to look at the connection between pot smoking and sex at the population level. To tease out the association, Eisenberg and his co-author Dr. Andrew Sun used survey data drawn from more than 50,000 Americans between the ages of 25 and 45.

The data comes from the National Center for Health Statistics at the CDC. Since that survey does not ask about homosexual encounters, the results only apply to straight men and women.

Eisenberg decided to do the study because more of his patients had been asking him if smoking marijuana could be contributing to their sexual difficulties. He had previously told them it might, and to abstain just in case. He was basing his assumptions on research about the negative vascular effects of cigarette smoking. In addition, some past studies and case reports have indicated heavy marijuana use may be associated with erectile dysfunction or depressed sperm count.

But now Eisenberg is rethinking his advice, at least for some patients.

“If somebody is using marijuana to help them for chronic back pain or something like that, there may be other interventions that we can think about targeting,” he said. “Rather than telling them they have to stop, otherwise their sex life is doomed.”

In her offices in Los Angeles and Portland, Oregon, Richmond has seen mixed results when her clients use marijuana. Some couples tell her that they have more sex when they use pot, but others have less sex.

She said those differences are probably attributable to how much pot someone smokes, instead of how often.

“Too much can lead to lethargy and really checking out, which does not facilitate [emotional] connection at all, and definitely doesn’t encourage sexual activity.”

Eisenberg cautioned against drawing unwarranted conclusions from the study, and cited the old statistical adage “correlation does not equal causation.”

“This doesn’t mean that if you want to have more sex you should start smoking marijuana,” he said. “That’s definitely not what this data supports.”

The study can’t explain what factors are driving the association between pot use and sex, said Dr. Igor Grant, chair of psychiatry and director of the Center for Medicinal Cannabis Research at the University of California San Diego.

He said one explanation is that people who use marijuana —or are willing to admit marijuana use in a survey —are more likely to report their sexual encounters, or remember more of them. Grant said marijuana users are also sensation seekers, and may be more driven to have sex.

“Drug use is one type of sensation-seeking behavior, and obviously sex is another,” Grant said.

Eisenberg agreed that personality and other behaviors could be a factor, but if that was the case, he would expect to see different results from different demographic groups. For example, young and single respondents might be more willing and able to engage in risky or sensation-seeking behavior, when compared to married people with children. But the results held across all categories, including race and ethnicity, educational level, income level, religious affiliation, and family status.

“For every group, the more marijuana use that they reported, the more sex they reported as well. So that was really interesting to me, and also made me think that there could potentially be some biologic explanation here,” Eisenberg said.

Richmond, the sex therapist, says she wouldn’t advise any client who doesn’t already smoke to start smoking marijuana as a sexual aid. But she said it could be reassuring to her pot-using clients to learn from the study that smoking pot doesn’t appear to decrease sexual activity.

“Individuals and couples look for additional ways to create novelty in the relationship and have fun, and that’s now a legal and accessible way to do it,” she said.

Medicinal marijuana is now legal in 29 states, and eight of them allow recreational use as well. (The District of Columbia allows both).

Dr. Eisenberg said that’s why learning more about the potential side effects of marijuana use is so critical.

The Delaware resident was diagnosed with hepatitis C more than two years ago, but she doesn’t qualify yet for the Medicaid program’s criteria for treatment with a new class of highly effective but pricey drugs. The recent approval of a less expensive drug that generally cures hepatitis C in just eight weeks may make it easier for more insurers and correctional facilities to expand treatment.

The drug, Mavyret, is the first to be approved by the Food and Drug Administration that can cure all six genetic types of hepatitis C in about two months in patients who haven’t previously been treated. Other approved drugs generally require 12 weeks to treat the disease and often aren’t effective for all types of hepatitis C.

In addition, Mavyret’s price tag of $26,400 for a course of treatment is significantly below that of other hepatitis C drugs whose sticker price ranges from about $55,000 to $95,000 to beat the disease. Patients and insurers often pay less, however, through negotiated insurance discounts and rebates.

“It certainly stands to reason that the continual march downwards on cost would lead to continual opening up of criteria,” said Matt Salo, executive director of the National Association of Medicaid Directors.

Hepatitis C is a viral liver infection spread through blood that affects an estimated 3.5 million people in the United States. It can take years to cause problems. Many baby boomers who contracted it decades ago before blood was screened for the virus don’t realize they have it until they develop liver disease. In addition, the growing heroin epidemic is adding to the problem as people become infected by sharing contaminated needles.

“Direct acting antiviral” therapies like Harvoni, a once-a-day pill introduced in 2014 that generally cured hepatitis C in 12 weeks, are much more effective than earlier treatments that required weekly interferon injections and multiple daily pills for nearly a year. But the newer regimens came at a price: $94,500, in Harvoni’s case.

State Medicaid programs, which cover a high proportion of people with hepatitis C, balked at the high prices, even with the 23 percent drug discount the programs typically receive. Many threw up roadblocks to limit drug approval until the disease was advanced. Some required people to be drug- and alcohol-free for six months or more before treatment would be approved.

Those moves prompted advocates to push for better access, in some cases filing suit to force the programs to cover more people.

Faced with a lawsuit in Delaware, the state Medicaid program began loosening up treatment criteria this year, and in January will begin approving enrollees regardless of the severity of their disease.

The state joins more than a dozen others that no longer (or never did) restrict hepatitis C treatment based on disease severity, said Kevin Costello, director of litigation at Harvard Law School’s Center for Health Law and Policy Innovation, which has been a key player in litigation in Delaware and other states.

It can’t happen soon enough, said Green, 58, who believes she contracted the disease 31 years ago when she suffered complications during childbirth and required a blood transfusion. Although her liver isn’t damaged, Green said, she’s suffered with abdominal and joint pain, weight loss and fatigue for decades, symptoms that doctors attribute to the hepatitis C virus.

“It’s been a difficult fight for us Medicaid patients,” she said.

People who are incarcerated face an even tougher battle to get treatment for hepatitis C. Roughly 17 percent of prisoners are infected with hepatitis C, compared with about 1 percent of the general population.

Prisons have a duty not to be deliberately indifferent to the medical needs of incarcerated people. Prisons don’t get the price discounts that the Medicaid programs have, and their budgets are fixed.

“Administrators have to make do with what is there,” said Dr. Anne Spaulding, an associate professor at Emory University’s public health school who has worked as a medical director in corrections and published research on hepatitis C among prisoners.

Lawyers in a handful of states are pursuing class action lawsuits to force prisons to provide hepatitis C treatment. Mavyret may make a difference, said David Rudovsky, a civil rights lawyer who’s litigating a class action lawsuit against the Pennsylvania Department of Corrections.

“Everyone recognizes that it’s going to make it easier to cover people,” he said.

People with regular private insurance may face some obstacles to coverage of hepatitis C, but coverage is typically less problematic. For example, Mavyret is one of seven hepatitis C drugs that are included in the 2018 national preferred formulary by Express Scripts, which manages the pharmacy benefits for 83 million people.

“The benefit to patients and payers is the additional competition, which brings down costs across the class, thus resulting in greater access and affordability,” said Jennifer Luddy, director of corporate communications at Express Scripts.

]]>https://ww2.kqed.org/stateofhealth/2017/10/04/hepatitis-c-drugs-lower-cost-paves-way-for-medicaid-prisons-to-expand-treatment/feed/0361327In the Battle Against Hepatitis C, the VA Takes the Leadhttps://ww2.kqed.org/stateofhealth/2017/09/21/in-the-battle-against-hepatitis-c-the-veterans-administration-takes-the-lead/
https://ww2.kqed.org/stateofhealth/2017/09/21/in-the-battle-against-hepatitis-c-the-veterans-administration-takes-the-lead/#commentsThu, 21 Sep 2017 21:57:55 +0000https://ww2.kqed.org/stateofhealth/?p=361087Junius Wilson likes to dress up when he goes in for his appointments at the Department of Veterans Affairs in Martinez. On a recent visit, the 67-year-old wore a black felt fedora and a sport coat. On his lapels, he wore a handful of military medals.

“All these are medals from Vietnam,” he said. “The three stars are for warriors … this one’s national defense.”

Wilson’s wife, Shelly Baker, sat beside him. She nudged him to show off one medal, which hung from a violet ribbon: a Purple Heart.

Wilson got that one because he was shot in the foot diving for cover during battle. He was in the infantry in South Vietnam and saw a lot of combat.

But Wilson, 67, isn’t here for that. He’s come for a monthly consultation with a specialized VA nurse who oversees his hepatitis C treatment.

The VA has implemented an aggressive effort to screen and treat all veterans under their care for the virus. Hepatitis C experts and advocates have praised the VA for its proactive approach, and say it should be a model for other government health programs, and even private insurers.

While about 1 percent of the U.S. population is infected with hepatitis C, veterans who use the VA have higher rates, 4.8 percent.

And Vietnam-era veterans like Wilson have an even higher rate, according to studies, VA data and physicians. Not all Vietnam veterans use the VA for health care, but among those who do, 7.5 percent have tested positive for the virus.

It’s unclear why, but there are several theories. One is simply the demographic overlap between Vietnam veterans and baby boomers. Members of the baby-boom generation have Hepatitis C infection rates five times higher than the average American. Hepatitis C, which is blood-borne, wasn’t even identified as a distinct virus until 1989, and the baby boomers grew up in an era when blood wasn’t screened, and before disposable needles were common in medical settings.

Some attribute the higher rate of Hepatitis C in Vietnam veterans to the injections they received before deploying: Troops sent to Vietnam were often vaccinated assembly-line style, with something called a jet injector. Instead of a needle, a jet injector uses high pressure to force a vaccine through the skin. Later, research showed that older versions of the device could transmit a hepatitis virus from person to person, and it’s no longer used in the military. While it is impossible to know if jet injectors spread the hep C virus, the VA has said it is “biologically plausible.”

Others point out that some veterans of this conflict used intravenous drugs while in Vietnam, or after returning to the U.S.

Hepatitis C is passed through the blood. About 3.5 million Americans have the virus, and it’s believed that half of them do not know it. After the discovery of the virus in 1989, screening of the blood supply didn’t begin until 1992.

The virus can lurk silently for years, slowly damaging the liver until symptoms appear, like pain, nausea and yellow skin. It kills more Americans than all other infectious diseases combined, including HIV. In 2014, the death toll reached an all-time high of 19,659.

VA pharmacist Diana Ruiz demonstrates how the VA tracks the status of veterans who have or had Hepatitis C. (Laura Klivans/KQED)

The VA’s push to find — and cure — every patient with the virus began three years ago.

This became feasible only when new hepatitis C medications came to market in late 2013. The new generation of drugs was a huge improvement over previous treatments. The older drugs required shots that were injected into the stomach, and the medications could have severe side effects: flulike symptoms and even mental health problems.

VA nurse Susan Bertilacchi-Green does not miss those days. A decade ago, the cure rate she observed from those older drugs was about 30 percent, she said.

“That was then,” she said. “Now, we have new medications that are nearly 100 percent effective. They have little to no side effects. You can be treated in as little as eight weeks, and there’s no shots anymore.”

Since 2014, the VA has cured 96,000 patients of hepatitis C.

Vietnam veteran Roman Walden is one of them. He thinks he got the virus while injecting heroin in Vietnam, a habit that continued when he returned home to Oakland. Years ago, he tried treatment on the old drugs, but had to stop due to serious side effects. When the new drugs came out, he was hesitant to try treatment again. But with reassurance from Bertilacchi-Green, and other veterans who’d been cured, he yielded. This time, the treatment worked.

Vietnam veteran Roman Walden has a renewed sense of life after being successfully cured of hepatitis C. (Laura Klivans/KQED)

“I was really amazed at that,” he said. “But then what really got me was how much it cost, and that made me feel like the president of the United States. They were spending this much money on me?”

Hepatitis C drugs have become notorious for their high cost. The wholesale price for a course of treatment can be over $100,000.

For Walden, that high price actually prompted him to take himself more seriously. He said the expense felt like an investment in him by the VA. After years on and off drugs, and in and out of jail, Walden, now 62, said he’s finally changing for the better. He said he no longer drinks or uses drugs, and is volunteering in hospitals, playing drums for patients.

The exorbitant prices of the new hepatitis C medications have led other health plans to hold off on treating every patient. Medi-Cal, for example, gives treatment priority to patients that show evidence of liver damage, or those who could spread the virus, like active drug users.

While the VA initially also had “treatment priorities,” this policy changed in 2016, after Congress stepped up with billions in funding, and pharmaceutical companies released new versions of drugs that were more competitively priced.

These days, the VA will treat anyone with any level of veterans benefits. For the veterans, the treatment is usually free. Some have a small co-pay for the entire course of treatment, with $33 as the maximum.

Health officials say hepatitis C could be eliminated as a public health risk by 2030, but it will happen only if other health plans copy the VA’s lead.

At the VA in Martinez, Bertilacchi-Green concludes the visit by showing Junius Wilson and his wife, Shelly Baker, his latest lab tests. Even though he still has a month to go on his medication, the virus is already undetectable in his blood.

Wilson and Baker smile at one another.

“We’re happy,” Baker said. “We still need him around for a long time.”

Additional reporting by Carrie Feibel.

]]>https://ww2.kqed.org/stateofhealth/2017/09/21/in-the-battle-against-hepatitis-c-the-veterans-administration-takes-the-lead/feed/4361087Physician-Assisted Suicide Has Been Legal in California for a Year. How’s it Going?https://ww2.kqed.org/stateofhealth/2017/06/07/physician-assisted-suicide-has-been-legal-in-california-for-a-year-hows-it-going/
https://ww2.kqed.org/stateofhealth/2017/06/07/physician-assisted-suicide-has-been-legal-in-california-for-a-year-hows-it-going/#commentsWed, 07 Jun 2017 21:00:57 +0000https://ww2.kqed.org/stateofhealth/?p=339498John Minor, a retired psychologist, epitomized the active Californian. A resident of Manhattan Beach, Minor ran long distances, bicycled, and hiked.

“He and my mom were both members of the Sierra Club,” said Jackie Minor of San Mateo, one of his daughters. “They went on tons of backpacking trips, you know — climbing mountains and trekking through the desert. He was just a very active person.”

But in the fall of 2014, Minor became ill with pulmonary fibrosis, a lung disease, and it was terminal. His family members said the disease slowly but surely eroded his quality of life. He became increasingly confined to his home, wracked by pain and violent fits of coughing, and suffered frightening episodes of choking and suffocation.

So two years after the diagnosis, on Sept. 15, 2016, John Minor sipped his last drink: apple juice laced with a lethal dose of medication his doctor had prescribed for him. He died surrounded by family members, at the age of 80.

“He laid down and went to sleep, and he was in a coma for about two hours and then he passed,” Jackie said. “It was very peaceful.”

California’s End of Life Option Act went into effect on June 9, 2016. The law created a process for dying patients to ask their doctors for a lethal prescription that the patients can then ingest privately, at home.

Since then, at least 500 Californians have received life-ending prescriptions, according to newly released data collected by Compassion and Choices, an advocacy group that promotes aid-in-dying laws nationwide.

The organization also estimated that about 500 health systems and 100 hospice organizations now participate in the California law.

In addition, 80 percent of insurance companies in the state are paying claims involved in the process, such as doctor visits and the prescription itself.

“What the numbers are showing is that the law is working incredibly well,” said Matt Whitaker, the organization’s director for California and Oregon. “It’s working as the lawmakers intended.”

But finding a doctor willing to prescribe the life-ending drugs isn’t always easy, in part because the state law allows doctors to opt out of prescribing — even when the hospital where they work participates in the law.

The decision to prescribe does not come easily for many physicians.

“Even if they’re in support of aid-in-dying, they don’t necessarily want to be the person identified as the ‘go-to’ person for aid-in-dying. Because that’s a very different implication,” said Dr. Elizabeth Dzeng, an assistant professor of hospital medicine at the University of California, San Francisco.

About three dozen UCSF patients have requested such assistance since last June.

“There is a certain stigma for being known as a physician who writes these prescriptions,” agreed Dr. Stephanie Harman, medical director of palliative care services at Stanford Health Care.

Harman decided to study how the law was playing out in the Stanford system. She tracked 13 Stanford patients who received prescriptions for lethal drugs, and learned that half of them initially couldn’t get the drugs from their own doctor.

“There is in the field, in medicine, a question of whether this is an ethical act for a physician,” she explained.

Other hospitals and health systems statewide are also trying to get a sense of what their terminal patients are experiencing. At the University of California, Los Angeles, about 20 patients have received prescriptions since the law went into effect — but only about half of them have gone on to take the meds, said Dr. Neil Wenger, a professor of medicine at UCLA and director of the UCLA Health Ethics Center.

Wenger helped develop a protocol for UCLA doctors, to guide them through the requirements of administering the medication to qualified terminally ill patients.

Among those he’s spoken with, there is a “whole range of preferences” regarding how and whether to participate in the law.

A “relatively small number” oppose the law outright, he said. Many others, however, believe patients should have access to aid-in-dying assistance, but don’t want to be involved in prescribing themselves, since it involves legal requirements, paperwork and conversations that can often be difficult.

“It raises a lot of feelings on the part of the doctor. It is something very, very different than what a doctor does — which is saving people,” Wenger explained. “And it’s complicated. It takes a whole lot of time.”

Dr. Catherine S. Forest is one doctor who has committed herself to taking that time, and assisting patients who want the prescription.

Forest practices family medicine in the Stanford health system. Since the law passed, she’s assisted five patients who came to her practice after their own doctors refused.

Forest says what’s happening now in California is reminiscent of what happened in the 1970s, when abortion became legal. Even among doctors who agreed with the Roe v. Wade decision, there was a reluctance to perform abortions.

“It takes awhile for people to train, to feel comfortable and to provide, and that was because it was not legal, and then it was legal. And there are very few instances where we do that in medicine,” Forest says.

Aid-in-dying, she says, is one of those challenging moments for doctors. She expects it will take time for California to catch up with other states, like Oregon, that have well-established training resources to help doctors learn the process.

John Minor wasn’t able to get a prescription at first. His doctors would not write the necessary prescription, so his family started scrambling.

“I started cold-calling just different hospitals and different departments within different hospitals,” Jackie said.

Ultimately, the family took the step of enrolling her father in a Kaiser Permanente insurance plan, and he received the prescription that he took last fall.

“Mentally, he was ready,” said his widow, Sherry Minor. “It was an easy day for him.”

]]>https://ww2.kqed.org/stateofhealth/2017/06/07/physician-assisted-suicide-has-been-legal-in-california-for-a-year-hows-it-going/feed/2339498Network of Scientists Works to Help Patients With Undiagnosed Diseaseshttps://ww2.kqed.org/stateofhealth/2017/04/27/network-of-scientists-works-to-help-patients-with-undiagnosed-diseases/
https://ww2.kqed.org/stateofhealth/2017/04/27/network-of-scientists-works-to-help-patients-with-undiagnosed-diseases/#respondThu, 27 Apr 2017 22:07:40 +0000https://ww2.kqed.org/stateofhealth/?p=323172WHITTIER, Calif. — Lynn Whittaker stood in the hallway of her home looking at the framed photos on the wall. In one, her son Andrew is playing high school water polo. In another, he’s holding a trombone.
The images show no hint of his life today: the seizures that leave him temporarily paralyzed, the weakness that makes him fall over, his labored speech, his scrambled thoughts.

Andrew, 28, can no longer feed himself or walk on his own. The past nine years have been a blur of doctor appointments, hospital visits and medical tests that have failed to produce answers.

“You name it, he doesn’t have it,” his mother said.

Andrew has never had a clear diagnosis. He and his family are in a torturous state of suspense, hanging their hopes on every new exam and evaluation.

Recently, they have sought help from the Undiagnosed Diseases Network, a federally funded coalition of universities, clinicians, hospitals and researchers dedicated to solving the nation’s toughest medical mysteries. The doctors and scientists in the network harness advances in genetic science to identify rare, sometimes unknown, illnesses.

At UCLA, one of the network’s sites, Andrew’s medical team would map his genetic makeup, then bring him in for a week of exams and consultations with specialists.

Writing A New Disease Encyclopedia

The Undiagnosed Diseases Network was founded in 2015 with a $43 million grant from the National Institutes of Health (NIH). Building on work already being done at NIH, the initiative expanded to include universities across the country: Duke, Columbia and Stanford are among the other sites. The goals are to provide answers for patients with mysterious diseases and to learn more about the disorders.
A proposal last month by President Donald Trump to cut the NIH budget by $5.8 billion could put the program in jeopardy.

Even with the best technology and the finest brains at work, progress is slow. Since its launch, the network has received nearly 1,400 applications on behalf of patients. It has accepted 545 for review so far. Just 74 of the cases have been diagnosed, including 11 at UCLA. Andrew Whittaker’s case is among many in progress.

It’s like battling “an unknown enemy,” said Euan Ashley, one of the principal investigators of the network’s Stanford University site. “That is a particular form of torment that other patients don’t have.”

A diagnosis can end families’ painful odyssey while helping physicians and scientists better understand rare diseases and human physiology, said Rachel Ramoni, former executive director of the network, which is based at Harvard University.

Researchers throughout the network use advanced medical technology. For example, to study patients’ gene expression and disease progression, they can make models using nearly transparent zebrafish, whose genetic structure is similar to that of humans. And scientists can conduct whole genome sequencing, which allows the medical team to read a patient’s DNA and identify changes that can reveal what may be causing a disease.

“We have powerful techniques to look at every gene that is being expressed as well as every gene that is inherited,” said Stanley Nelson, one of UCLA’s principal investigators and the lead doctor on Andrew’s case. “This is an example of true precision medicine.”

Nelson said the network can examine all known genes — not just the ones believed to have mutations that cause diseases. Doing that can lead to the discovery of new illnesses.

“Part of what we have to do is keep building that library, that encyclopedia of what gene and what gene mutations cause what symptoms,” Nelson said. “It’s just incomplete at this moment.”

Already the work is helping patients and their families come to terms with their illnesses. In one case, at Stanford, a toddler was diagnosed with two rare diseases, including a connective tissue disorder called Marfan Syndrome, after doctors conducted a form of sequencing that looks for changes in coded genetic segments known as exons.

“A lot of times your ability to be diagnosed depends on who is in the room,” Ramoni said. “And what we are doing with the network is we are expanding exponentially the number of people in the room.”
Doctors at one institution might think their patient is a unique case, only to learn that colleagues elsewhere have a patient with a similar illness. But even when diseases are diagnosed or gene mutations are discovered, treatments may still not be available.

A Life-Changing Mystery

Andrew Whittaker’s odyssey began one afternoon at age 19, when he started trembling and couldn’t speak. Doctors suspected he was suffering from anxiety and prescribed medication to control it. But Andrew said he continued to have “episodes,” during which everything just went blank.

Andrew also started losing his balance and falling off his bicycle. The family visited several hospitals. Doctors discovered that the receptors in his brain were malfunctioning and that he lacked sufficient dopamine, a chemical compound in the body responsible for transmitting signals between nerve cells. As a result, Andrew has some symptoms similar to those of Parkinson’s disease. Doctors also confirmed he was having seizures.

Still, Andrew’s symptoms didn’t add up to any known disease.

One afternoon last fall at precisely noon, as Andrew sat propped up on the living room couch, Lynn’s phone alarm sounded, signaling it was time for his medication. Lynn pried open Andrew’s hand, which was clenched into a fist, and dropped in the pills.

To keep Andrew from falling, the family has lowered his bed and removed carpet from the house. They also bought him a wheelchair. Their precautions don’t always work. One morning, Lynn was in the kitchen when she heard a crash. “I ran in there and he’s laid flat on his back,” she said.

Andrew is close to his mom. But he also gets frustrated. He can’t shower or dress without her help. He’s had to give up the things he loved to do: printing T-shirts. Skateboarding. Shooting short films. He’s lost friends and can’t imagine dating anymore.

“Girlfriends? Forget about it,” he said, his face twitching as he talks. “They want a guy who can do stuff for them, not the other way around.”

Running The Medical Gauntlet

On a Monday morning in late January, Andrew and his parents were in an exam room at UCLA. Lynn teased her son, saying she was going to put him in a freezer until doctors figured out what was wrong.
“Then we’ll pull you back out again,” she said, smiling.

“I’ll never get pulled out,” Andrew responded.

“Yes, you will,” she said. “You will.”

Nelson, Andrew’s main doctor, walked into the room. He told Andrew he’d read through the medical records. “We’re going to try to figure you out.”

The work Nelson does is personal. His teenage son, Dylan, has Duchenne muscular dystrophy, a genetic disorder that causes muscle degeneration and weakness. Nelson knows his son’s disease will eventually take his life, but he said having a diagnosis makes all the difference.

“My heart very much goes out to the families that don’t even get an adequate diagnosis,” he said.
Nelson suspects that Andrew’s disease is genetic as well.

He asked the Whittakers to describe their son’s journey and he conducted a short physical exam, asking Andrew to push against his hand and touch his own nose. Andrew trembled and his shoulders tensed, but he did it.

The rest of the week, Andrew underwent several other diagnostic tests, including a muscle biopsy, an EEG, MRI and a lumbar puncture. He remained upbeat, though running the medical gauntlet clearly wore him out. He also met with UCLA specialists in brain degeneration and muscle and nerve disorders.

At week’s end, Nelson sat down with the family to explain what he’d found. He had reviewed Andrew’s genome and compared it with that of both parents. Andrew had one copy of a defective gene that leads to Parkinson’s but the genome sequencing didn’t show a second copy, without which it could not be Parkinson’s.

He also explained that Andrew’s illness was clearly progressive and that his brain was shrinking, making it harder for him to process language and information. Nelson said he still didn’t have a diagnosis — he believed it was a brand-new disease.

Nelson planned to continue poring over the test results, conducting additional exams and communicating with others in the network. He also is analyzing Andrew’s muscle, skin and blood to see whether any mutated gene is expressed abnormally.

Even in the absence of a clear diagnosis, Nelson said, rare diseases like Andrew’s help educate scientists and may help other patients. “These are the people we as a society will owe a great debt of gratitude,” he said. “They are effectively donating their lives to this process.”

Lynn Whittaker was disappointed. “We are still left with just hope that they will come up with something,” she lamented. “What else do we have?”

Andrew said his relatives have asked if he’s scared the doctors will find something. “I’m more scared if they don’t,” he replied.

]]>https://ww2.kqed.org/stateofhealth/2017/04/27/network-of-scientists-works-to-help-patients-with-undiagnosed-diseases/feed/0323172Hype Offers Hope — and Risks — to Cancer Patientshttps://ww2.kqed.org/stateofhealth/2017/04/27/hype-offers-hope-and-risks-to-cancer-patients/
https://ww2.kqed.org/stateofhealth/2017/04/27/hype-offers-hope-and-risks-to-cancer-patients/#respondThu, 27 Apr 2017 17:04:13 +0000https://ww2.kqed.org/stateofhealth/?p=323065After Michael Uvanni’s older brother, James, was diagnosed with a deadly form of skin cancer, it seemed as if everyone told the family what they wanted to hear: Have hope. You can beat this, and we are here to help.

The brothers met with doctors at a half-dozen of the country’s best hospitals, all with impressive credentials that inspired confidence.

Michael Uvanni was in awe when he visited the University of Texas MD Anderson Cancer Center in Houston, one of the world’s most respected cancer hospitals. It was like seeing the Grand Canyon, said Uvanni, 66, of Rome, N.Y. “You never get used to the size and scope.”

Even the MD Anderson logo on buses and buildings — with “Cancer” crossed out in red, above the words “Making cancer history” — made the family’s battle seem winnable.

“I thought they were going to save him,” said Uvanni, an interior designer.

Patients and families are bombarded with the news that the country is winning the war against cancer. The news media hypes research results to attract readers. Drug companies promise “a chance to live longer” to boost sales. Hospitals woo paying customers with ads that appeal to patients’ fears and hopes.

“I’m starting to hear more and more that we are better than I think we really are,” said Dr. Otis Brawley, chief medical officer at the American Cancer Society. “We’re starting to believe our own bullshit.”

The consequences are real — and they can be deadly. Patients and their families have bought into treatments that either don’t work, cost a fortune or cause life-threatening side effects.

“We have a lot of patients who spend their families into bankruptcy getting a hyped therapy that [many] know is worthless,” Brawley said. Some choose a medicine that “has a lot of hype around it and unfortunately lose their chance for a cure.”

Although scientists have made important strides in recent years, and many early-stage cancers can now be cured, most of those with advanced cancer eventually die of their disease.

For Uvanni, hope gave way to crushing disappointment when his brother’s health declined and he died from metastatic melanoma in 2014.

“You get your hopes up, and then you are dropped off the edge of a cliff,” said Uvanni. “That’s the worst thing in the world.”

Caregivers like Uvanni can suffer prolonged grief and guilt if their loved ones are riddled with side effects and don’t survive as long as the family expected, noted Holly Prigerson, co-director of the Center for Research on End-of-Life Care at Weill Cornell Medical College.

For decades, researchers have rolled out new cancer therapies with great fanfare, announcing that science has at last found a key to ending one of the world’s great plagues, said Dr. Vinay Prasad, an assistant professor of medicine at Oregon Health & Science University. When such efforts fail to live up to expectations, the cancer world simply moves on to the next big idea.

Hyping early scientific results — based on lab tests or animal studies — can attract investors that allow researchers to continue their work. Positive results can lead biotech firms to be bought out by larger drug companies.

“It’s in the interest of almost every stakeholder in the health system to be optimistic about these therapies,” said Dr. Walid Gellad, co-director of the Center for Pharmaceutical Policy and Prescribing at the University of Pittsburgh.

Of course, there is plenty of money to be made.

The U.S. spent nearly $88 billion treating cancer in 2014, with patients paying nearly $4 billion out-of-pocket, according to the American Cancer Society Cancer Action Network. Spending on cancer, a disease that most afflicts the aging, is predicted to soar as people live longer.

“While many people are trying to make patients’ lives healthier and longer and better, there are others that are exploiting their vulnerability,” said Dr. Leonard Saltz, chief of the gastrointestinal oncology service at New York’s Memorial Sloan Kettering Cancer Center.

Others argue that the excitement about cancer research is justified. A spokeswoman for the Pharmaceutical Research and Manufacturers of America, an industry group, said cancer patients have good reason for optimism.

“We continue to see great strides in identifying the genetic mutations and related factors that can drive the seemingly random formation of abnormal cells in cancer,” spokeswoman Holly Campbell said in a statement. “In the last decade, we’ve seen a number of scientific advances transform the landscape of many cancers.”

Promises To Cure Abound

Even the country’s top scientists sometimes get carried away.

In 1998, Nobel laureate James Watson — who co-discovered the structure of DNA — told The New York Times that scientists would “cure cancer in two years” using drugs that block tumor blood supplies. At that time, the drugs had succeeded only in mice.

In 2003, the director of the National Cancer Institute, Dr. Andrew von Eschenbach, announced a goal of “eliminating suffering and death due to cancer by 2015” by better understanding tumor genetics.

Last year, when President Barack Obama announced the Cancer Moonshot, which aims to accelerate and better coordinate research, he said, “Let’s make America the country that cures cancer once and for all.”

In a recent interview, von Eschenbach acknowledged he didn’t communicate his goal well.

“We all fall into that trap,” said von Eschenbach, now a senior fellow at the Milken Institute, a health and public policy think tank. “We’re offering what we have, but making it appear that it’s more than what it is.”

It’s easy to see how patients’ hopes are raised, said Timothy Turnham, former executive director at the Melanoma Research Foundation, an advocacy group. Researchers are frequently overly enthusiastic about early discoveries that have little chance of leading to a new drug.

“There is a disconnect between what researchers think is statistically significant and what is really significant for patients,” Turnham said. “Patients hear ‘progress,’ and they think that means they’re going to be cured.”

A Marketing Blitz

Uvanni said his brother’s experience was nothing like the sunny images in TV commercials, in which smiling cancer patients hug their grandchildren, hike in the mountains and lead dance classes.

A TV commercial for the Bristol-Myers Squibb drug Opdivo projects the words “a chance to live longer” on the side of skyscrapers, as a captivated crowd looks on. In much smaller type, a footnote reveals that lung cancer patients taking Opdivo lived just 3.2 months longer than others.

A TV ad for Merck’s Keytruda features reassuring images of a smiling, healthy patient hugging her family — not fighting for breath or struggling to walk. Although the commercial notes that the people in the ad are portrayed by actors, the commercial claims the drug provides “a chance for a longer life. It’s Tru.”

“Your heart sinks when you see those ads,” Uvanni said. Seeing the family depicted in the ad, he said “makes you wonder if they’re going down the same path that we did.”

The Keytruda ad notes that 71 percent of patients given the drug were alive “at the time of patient follow-up,” compared with 58 percent of those who received chemotherapy. The ad doesn’t mention that the “time of follow-up” was 11 months.

“It’s not false; it’s just incomplete,” said pharmacist Harold DeMonaco, a visiting scientist at the Massachusetts Institute of Technology in Boston. “They don’t give patients or the patients’ family enough information to make a reasonable decision.”

In an interview, Merck senior vice president Jill DeSimone said that the company aims to be responsible with its advertising, noting that the Keytruda ad reminds patients to talk to their doctors. “The physician is the ultimate decider on treatment,” DeSimone said.

In a statement, Bristol-Myers’ senior vice president Teresa Bitetti said that Opdivo ads play “an important role in educating patients about new treatment options and fostering informed conversations between patients and their doctors.”

Hospitals also have drawn criticism for overstating their success in treating cancer. In 1996, Cancer Treatment Centers of America, a for-profit chain, settled allegations from the Federal Trade Commission that “they made false and unsubstantiated claims in advertising and promoting their cancer treatments.”

The company’s current commercials — dozens of which are featured on their website — boast of offering “genomic testing” and “precision cancer treatment.”

The commercials don’t tell patients that these tests — which aim to pair cancer patients with drugs that target the specific mutations in their tumors — are rarely successful, Prasad said. In clinical trials, these tests have matched only 6.4 percent of patients with a drug, according to Prasad’s 2016 article in Nature. Because these drugs only manage to shrink a fraction of tumors, Prasad estimates that just 1.5 percent of patients actually benefit from precision oncology.

In a statement, Cancer Treatment Centers of America said, “We use national media to help educate cancer patients and their families about the latest diagnostic tools and treatment options. … All of our advertising undergoes meticulous review for clinical accuracy as well as legal approval to ensure we tell our story in an informative and responsible manner, and in compliance with federal guidelines.”

Spending on ads for hospitals that treat cancer soared 220 percent from $54 million in 2005 to $173 million in 2014, according to a 2016 article in JAMA Internal Medicine. Ads for Cancer Treatment Centers of America accounted for nearly 60 percent of all total cancer center advertising.

Targeting Melanoma

For more than a decade, the Food and Drug Administration approved no new treatments for metastatic melanoma. Patients typically died within a year of diagnosis.

Since 2011, however, the FDA has approved 11 new treatments, including several immunotherapies, which aim to harness the immune system to fight cancer. Last year, doctors leading a clinical trial announced that the median survival of patients taking the drug Keytruda had grown to two years. Forty percent of patients were alive three years later, according to the clinical trial, presented at the American Society of Clinical Oncology.

Researchers have tested immunotherapies against a variety of tumors, leading to approvals in lung cancer, kidney cancer, bladder cancer and others.

Such success has led doctors to label cancer immunotherapy as a “game changer.” N­­ewspapers and magazines call it a “breakthrough.” And hospitals laud them as “a miracle in the making.”

Yet these treatments — which were initially assumed to be gentler than chemotherapy — can provoke fatal immune system attacks on the lungs, kidneys, heart and other organs.

And there are no approved immunotherapies for tumors of the breast, colon, prostate and pancreas.

Only about 10 percent of all cancer patients can expect to benefit from immunotherapy, Prasad said.

Uvanni’s brother — who tried immunotherapy, as well as a number of other approved and experimental treatments — survived 3½ years after his diagnosis. That might lead many oncologists to describe his story as a success.

Uvanni sees no reason to celebrate. He wanted more than short-term survival for his brother.

“I thought we were going to have a treatment where we’d at least have a good block of quality time,” Uvanni said.

But treatments meant to control the cancer only made him sick. Some caused flu-like symptoms, with fever, chills and shakes. Others left him nauseated, unable to eat or move his bowels. Others caused dangerous infections that sent him to the emergency room.

“I hope that if something like that happens to me,” Uvanni said, “I would be strong enough to say no to treatment.”

]]>https://ww2.kqed.org/stateofhealth/2017/04/27/hype-offers-hope-and-risks-to-cancer-patients/feed/0323065Hundreds of Babies Reported Harmed by Homeopathic Producthttps://ww2.kqed.org/futureofyou/2017/02/24/hundreds-of-babies-reported-harmed-by-homeopathic-product/
Mon, 27 Feb 2017 21:37:39 +0000https://ww2.kqed.org/stateofhealth/?p=297749Case 7682299: Aug. 1, 2010. A mother gives her toddler three homeopathic pills to relieve her teething pain. Within minutes, the baby stops breathing.
A review of FDA records shows that some babies who were given Hyland’s teething products had repeated seizures, became delirious or turned blue and died. (Photo from Read More …

]]>297749CRISPR Patent Ruling: 3 Different Takeshttps://ww2.kqed.org/futureofyou/2017/02/23/crispr-patent-ruling-3-different-takes/
Fri, 24 Feb 2017 19:29:57 +0000https://ww2.kqed.org/stateofhealth/?p=297429So last week the feds may have dashed UC Berkeley’s billion-dollar dreams when it decided not to give the university patent rights over CRISPR-Cas9, a revolutionary gene-editing technology.

]]>297429Talking is Easy for Therapists, Except When It’s About Guns. Veterans Want to Teach Them How.https://ww2.kqed.org/stateofhealth/2017/02/01/talking-is-easy-for-therapists-except-when-its-about-guns-veterans-want-to-teach-them-how/
https://ww2.kqed.org/stateofhealth/2017/02/01/talking-is-easy-for-therapists-except-when-its-about-guns-veterans-want-to-teach-them-how/#commentsWed, 01 Feb 2017 09:00:16 +0000http://ww2.kqed.org/stateofhealth/?p=274976Jay Zimmerman got his first BB gun when he was seven, and his first shotgun when he was ten.

“Growing up in Appalachia, you look forward to getting your first firearm probably more so than your first car,” he says.

His grandfather taught him to hunt squirrel and quail. Zimmerman, who lives in Tennessee, says pretty much everyone he knows has a gun. It’s just part of the culture.

“When I went into the military, that culture was reinforced,” he says. “Your weapon is almost another appendage. It’s part of who you are.”

Zimmerman was a medic in the army in the late 1990s and early 2000s. He served in Bosnia, Africa, and the Middle East. Since he came home, he’s struggled with PTSD and depression. It reached a crisis point a few years ago, when his best friend — the guy who had saved his life in a combat zone — killed himself. Zimmerman decided his time was up too.

“I decided that I would have one more birthday with my daughter, one more Christmas with my daughter,” he says. “I had devised my own exit strategy for sixteen February, 2013.”

But then he bumped into a woman who used to ride the same school bus when they were kids. His exit date came and went. They’re married now.

Zimmerman is a peer counselor at the Mountain Home VA Medical Center in Johnson City, Tennessee. He also travels to conferences all over the country, sharing his story with therapists and with other vets. He tries to set an example that it’s okay to ask for help. Even today, if he’s not doing well, he disassembles his guns and stores them separately from ammunition, so he can’t make any rash decisions. If things get really bad, Zimmerman has a special arrangement with a few friends.

“I call them and say, ‘Look, I’m feeling like it’s not safe for me to have firearms in my home. Can you store them for me for a couple days till I feel like I’m OK to have them back?'”

Jay Zimmerman (right) was an army medic. He poses with his dad before he deployed to Bosnia in 1998. (Photo Courtesy of Jay Zimmerman)

Suicide is an impulsive act. Nearly half the people who survive an attempt say the time between their first thought of suicide and the attempt itself was less than 10 minutes. But the method can mean the difference between life and death: people who take pills have time to change their minds. Not with guns.

About 70 percent of veterans who commit suicide do so with a gun, which prompted President Barack Obama to order the VA to talk to vets about gun safety and storage options like the ones Zimmerman uses.

But here’s the trouble: Most therapists aren’t gun people. They don’t know how to talk about guns. And so they don’t.

“One obvious reason for that is that no one has taught them how,” explained Megan McCarthy, National Deputy Director in the Office for Suicide Prevention at the Department of Veterans Affairs.

McCarthy was invited to speak recently at a suicide-prevention conference in San Francisco for therapists who work with vets.

“How many of you would say you feel really comfortable having a conversation with any of the people you work with about limiting access to all lethal means?” she asked the roomful of therapists.

Hardly anyone raised their hand.

“Okay, so that’s why we’re here today,” she said.

Researchers recommend starting with a field trip to a shooting range. There, therapists can learn about different kinds of firearms, as well as gun locks, and get an introduction to gun culture.

When counseling vets, therapists have to ask more questions and be less directive, according to McCarthy.

“We often conceive of ourselves as experts, as people who impart information to clients,” she said. But with vets, “it may take time to build trust. Telling them what to do the first time you’ve met them is probably not going to be a very effective approach.”

Therapists learn how to talk to vets about guns at a conference in San Francisco.

McCarthy presented a case study at the conference: A 28-year old army veteran who fought in Iraq told his VA psychiatrist that he had an argument with his girlfriend last week. He drove to an empty parking lot and sat with his loaded handgun in his lap, intending to kill himself.

He didn’t do it. A week later, the man told his psychiatrist things were still tense with his girlfriend. But he didn’t want to talk about suicide or storing his gun.

McCarthy asked the clinicians in the audience what they would do next, if they were this man’s psychiatrist.

“Why did he not do it? That would be my question,” said one therapist.

“I’d say, would you be willing to talk more about that?” said another.

“I would want to see this individual again, within the same week,” said a third. “I believe in strong intervention.”

Jay Zimmerman, the former army medic and peer counselor, stood up. He told them they’re all wrong.

“Chances are the reason he’s not talking to you is because he’s afraid he’s going to lose his gun, that he carries pretty much all the time,” he said. “My buddies are the same way, we all carry all the time.”

Zimmerman said the vet in the case study would rather talk to someone like him than someone in a white coat.

“If he’s got that good relationship with me as a peer, as a buddy, he’s probably already called me and talked to me,” he explained.

The takeaway for psychologists at the San Francisco conference, McCarthy said, is that sometimes their role is not to intervene, but to be a facilitator, someone who can connect vets with peer counselors like Zimmerman, or suggest they talk with a buddy, not always a professional.

]]>https://ww2.kqed.org/stateofhealth/2017/02/01/talking-is-easy-for-therapists-except-when-its-about-guns-veterans-want-to-teach-them-how/feed/3274976jay-zimmerman-in-uniform-and-dadJay Zimmerman right) was an army medic. He poses with his dad before he deployed to Bosnia in 1998.afsp-vaconference-audienceTherapists learn how to talk to vets about guns at a conference in San Francisco.Was the EpiPen Hack Ethical?https://ww2.kqed.org/futureofyou/2017/01/23/was-the-epipen-hack-ethical/
Mon, 23 Jan 2017 20:48:07 +0000https://ww2.kqed.org/stateofhealth/?p=286844Should we be free, as individuals, to make and take our own medicine at home? Who’s responsible if we get hurt or die? Do we have to right to do what we wish with our bodies in the interests of survival, healing and self-care? For years, such questions have been Read More …

By the drugmakers’ calculations, the markets for such diseases weren’t big enough to bother with.

But lucrative financial incentives created by the Orphan Drug Act signed into law by President Ronald Reagan in 1983 succeeded far beyond anyone’s expectations. More than 200 companies have brought almost 450 “orphan drugs” to market since the law took effect.

Yet a Kaiser Health News investigation shows that the system intended to help desperate patients is being manipulated by drugmakers to maximize profits and to protect niche markets for medicines already being taken by millions. The companies aren’t breaking the law but they are using the Orphan Drug Act to their advantage in ways that its architects say they didn’t foresee or intend. Today, many orphan medicines, originally developed to treat diseases affecting fewer than 200,000 people, come with astronomical price tags.

And many drugs that now have orphan status aren’t entirely new. More than 70 were drugs first approved by the Food and Drug Administration for mass market use. These medicines, some with familiar brand names, were later approved as orphans. In each case, their manufacturers received millions of dollars in government incentives plus seven years of exclusive rights to treat that rare disease, or a monopoly.

Drugmakers of popular mass market drugs later sought and received orphan status for the cholesterol blockbuster Crestor, Abilify for psychiatric conditions, cancer drug Herceptin, and rheumatoid arthritis drug Humira, the best-selling medicine in the world.

More than 80 other orphans won FDA approval for more than one rare disease, and in some cases, multiple rare diseases. For each additional approval, the drugmaker qualified for a fresh batch of incentives. Botox, stocked in most dermatologists’ offices, started out as a drug to treat painful muscle spasms of the eye and now has three orphan drug approvals. It’s also approved as a mass market drug to treat a variety of ailments, including chronic migraines and wrinkles.

Altogether, KHN’s investigation found that about a third of orphan approvals by the FDA since the program began have been either for repurposed mass market drugs or drugs that received multiple orphan approvals.

“What we are seeing is a system that was created with good intent being hijacked,” said Bernard Munos, a former corporate strategy advisor at drug giant Eli Lilly and Co. who reviewed the KHN analysis of several FDA drug databases. It’s “quite remarkable that it has gone on for so long.”

And the proportion of new drugs approved as orphans has ballooned. In 2015, 21 orphan drugs were approved, accounting for 47 percent of all new medicines, up from just 29 percent in 2010; in 2016, nine more orphans won approval, 40 percent of the total.

When a drugmaker wins approval of a medicine for an orphan disease, the company gets seven years of exclusive rights to the marketplace, which means the FDA won’t approve another version to treat that rare disease for seven years, even if the brand name company’s patent has run out. The exclusivity is compensation for developing a drug designed for a small number of patients whose total sales weren’t expected to be that profitable.

But the exclusivity is a potent pricing tool. Drugmakers can charge whatever they want by shielding their medicine from competition. The market exclusivity granted by the Orphan Drug Act can be a vital part of the protective shield that companies create. What’s more, manufacturers can return to the FDA with the same drug again and again, each time testing the drug against a new rare disease.

Critics have assailed drugmakers in the past for gaming the orphan drug approval process. But the extent to which companies have been winning approval for drugs that aren’t what advocates call “true orphans” hadn’t been documented until the Kaiser Health News investigation.

Munos said he was “shocked” by the sheer number of mass market drugs being repurposed as well as those approved multiple times.

Even agency officials said they weren’t aware of the scope of the issue. After reviewing KHN’s findings for two weeks, Dr. Gayatri Rao director of the FDA’s Office of Orphan Products Development said she “appreciated the work” and expressed interest in studying how often drug companies are “repurposing” a drug for a new rare disease, or taking “multiple bites of the apple.”

“We are going to look into this,” she said, adding that she could consider a regulatory change.

Rao pointed out that the “repurposing” of drugs does have scientific and patient benefits. For example, cancer drugs approved for one type of malignancy can be tested and approved for others. Gleevec, a drug that revolutionized the treatment of chronic myeloid leukemia, now has nine orphan approvals.

But in a 2015 commentary published in the American Journal of Clinical Oncology, Dr. Martin Makary at the Johns Hopkins University School of Medicine focused on cancer drugs including Gleevec, arguing that the drug was never meant to serve an orphan population. Instead, Makary and his team wrote, drugmakers purposely identify small patient populations to gain additional approvals — a process he described as “salami slicing.”

“By salami slicing the disease into subgroups, it allows them to get the orphan drug approval with all the government benefits and even some of the subsidies,” Makary said. The prices of such medications often rise because they have seven years without competition for a new set of patients, Makary added.

The FDA has taken a different view of repurposing.

“We always talked about how we permit the second bite of the apple, third bite of the apple, as one small way to incentivize repurposing,” Rao said, noting that industry and patient groups have been pressing the FDA for even stronger incentives. “Now, all of sudden, it seems like, wow, this practice may be driving up prices.”

Novartis, which owns Gleevec, said in an email statement that the company is advancing research and following the science to “bring the right treatments to the right patients based on unmet need, not the size of the patient population.”

Two KHN reporters spent six months analyzing data and talking to lawmakers, patients, advocates, doctors and companies to understand how the FDA’s orphan drug program has evolved amid a national uproar over soaring drug prices. President-elect Donald Trump vowed on the campaign trail to bring down prescription drug prices and during a Jan. 11 press conference said the drug industry is “getting away with murder.”

The investigation examined how drugmakers use the law to their advantage — often with the guidance of former FDA officials — and have made the development of medicines that were once thought to be business backwaters into one of the pharmaceutical industry’s hottest sectors.

Orphan drugs now account for seven of the 10 top-selling drugs of any kind, ranked by annual sales, according to EvaluatePharma.

“Orphans are wicked hot,” said Dr. Tim Coté, a former FDA official who now runs a consulting firm that advises drugmakers on orphan drugs.

No one disputes that orphan drugs have helped or saved hundreds of thousands of patients suffering from debilitating or even fatal rare diseases. Exactly how many is difficult to estimate because the FDA doesn’t track such information.

And drug industry officials say companies should be rewarded, not punished, for making those treatments possible and for pursuing new drugs that aren’t always an economic success.

Research and development is “long, costly, risky,” said Anne Pritchett, vice president, policy and research at industry lobbying group PhRMA. “When you look at cystic fibrosis it was 25 years to the development of an effective therapy … I think we would be concerned about anything that would undermine the current [orphan drug] incentives.”

Former U.S. Rep. Henry Waxman, D-Calif., a champion of the 1983 Orphan Drug Act takes a different view.

“The Orphan Drug Act has been a great success because many people with diseases that affect very few people now have drugs available to them,” Waxman said. “But it’s been in some ways turned on its head when it becomes the basis of manipulating the system for the drug company to make much more money than they would in an open, competitive market.”

Booming Business

On a late summer day, Tim Coté sat in a corner office of his Sandy Spring, Md., consulting firm, Coté Orphan. He leaned into his computer microphone to dispense insider knowledge about the orphan drug approval process on a webcast hosted by FDAnews, a trade news organization. Listeners paid about $300 a head, but Coté said he wasn’t paid for doing it.

The FDA is more flexible in evaluating drugs for rare diseases, he said, explaining that “about half of them get through with just one pivotal clinical trial. Not so for common diseases.” The FDA, citing a report from the National Organization for Rare Disorders, said about two-thirds of orphan drugs were approved with one adequate and well-controlled trial with supportive evidence. It typically requires two or three such trials to approve a mass market drug.

Coté also told the webinar audience that clinical trials for orphan drugs are usually smaller and the approval is a “different scientific and regulatory experience.”

Coté knows his stuff. He was Rao’s immediate predecessor as chief of the FDA’s Office of Orphan Products Development. It’s not unusual for government officials to leave FDA and other regulatory agencies and obtain jobs as consultants or industry executives.

Coté’s website, headlined “The Inside Track,” notes that he oversaw applications that led to the approval of at least 150 orphan drugs when he was at the FDA and that his firm is now the largest submitter of orphan drug applications.

“We write the entire application,” the website for Coté’s company notes, adding that his staff of 25 includes regulatory scientists with deep knowledge and experience in FDA’s “unwritten rules” regarding orphan drugs.

Many of Coté’s more than 300 clients are small biotech companies begun by researchers or even passionate parents who found investment backing. Parents Ilan and Annie Ganot, for example, started Solid Biosciences to find treatments and potentially a cure for their son with Duchenne muscular dystrophy.

Coté guides them through the regulatory process since most don’t have the expertise. He can offer his expertise and develop an application that makes it easier for the FDA to designate and approve the drug.

“When you make the FDA smile, the value of your asset goes up. And that’s how the game is played,” he said in an interview, adding quickly, “It’s really not a game because people’s lives are what is in balance.”

Coté and other ex-FDA officials play a vital role in helping drugmakers choose rare disease targets and get through the FDA approval process.

A small cottage industry has grown around the Orphan Drug Act. Dr. Marlene Haffner, who preceded Coté in the FDA’s orphan office, started her own consulting firm, too, to advise small and large companies on orphan drug applications. A third company is Camargo Pharmaceutical Services, led by industry veterans and former FDA officials, which advises companies focused on repurposing drugs for orphan approval. The firm tries “to be in front of the FDA a lot — three to four times a month,” said Jennifer King, Camargo’s director of marketing. Fees for consulting on orphan drugs industry wide range from $5,000 to $100,000, depending upon what services are provided, Coté said.

Getting through the orphan approval process involves a series of steps.

First, drugs must be designated by the FDA as potential candidates for approval. A company has to demonstrate that its drug is a promising treatment for a disease that affects fewer than 200,000 patients. If the FDA agrees and makes the formal designation, financial incentives kick in, including a 50 percent tax break on research and development (R&D) and access to federal grants.

When drugs get orphan designation, companies often reap other financial rewards. Shares in publicly traded companies often rise on the news — sometimes soaring as high as 30 percent. That happens, in part, because orphans have a track record of being approved at much higher rates than drugs for common diseases.

The 50 percent R&D tax credit pays off, too. In 2012, one of the biggest orphan drug companies, BioMarin, received $32.6 million from a combination of federal and state of California tax credits. BioMarin spokeswoman Debra Charlesworth confirmed that the orphan credit made up the “vast majority” of that deferred tax benefit. She also noted that credit “has successfully fueled an industry that didn’t previously exist” and led to more rare disease research.

Industry-wide, orphan drug tax credits cost the federal government $1.76 billion in fiscal 2016 — roughly what President Barack Obama asked Congress to spend to fight the Zika virus before a $1.1 billion expenditure was approved. And, because so many orphan drugs are under development, the U.S. could grant nearly $50 billion in tax credits from 2016 to 2025, estimates the Treasury Department.

There’s a lot of creativity behind figuring out how to make a drug an orphan.

In Coté’s webinar and in multiple interviews, he described many ways companies can win orphan status. They can test their drugs on children with adult diseases, such as schizophrenia, or find drugs for ailments like malaria that are uncommon in America.

“African sleeping sickness: horrible problem in Africa but not here, not in the U.S.,” Coté told his webinar audience. “So a drug development effort that was aimed toward some of these tropical diseases can actually get all the benefits of the Orphan Drug Act.”

Another popular strategy is to create “follow-on drugs” that represent incremental steps forward.

About 30 percent of Coté’s clients are companies looking to improve upon some other orphan drug “which just made billions and billions,” he said in an interview.

Repurposing an already approved drug is another strategy his firm promotes. In a video posted on his website in July, Coté explained the advantages for companies that can move directly into a clinical trial without much preparatory work because the drug’s safety has already been demonstrated.

“All you gotta do is establish that the product can work in this new orphan indication,” he said, adding tips on how to do it and still make money.

‘That Is Not A True Orphan Drug’

Turning mass market drugs into orphans has been a familiar path for some of the most popular drugs ever discovered.

AbbVie’s Humira is the best-selling drug in the world, and most of its sales are in the U.S. where revenue reached $7.6 billion through the third quarter of 2016 and $11.8 billion worldwide, according to the company’s latest financial report.

Humira was approved by the FDA in late 2002 to treat millions of people who suffer from rheumatoid arthritis. Three years later, AbbVie asked the FDA to designate it as an orphan to treat juvenile rheumatoid arthritis, which they told the FDA affects between 30,000 and 50,000 Americans. That pediatric use was approved in 2008, and Humira subsequently was approved for four more rare diseases, including Crohn’s and uveitis, an inflammatory disease affecting the eyes.

The ophthalmologic approval would extend the market exclusivity for Humira for that disease until 2023. When asked why AbbVie sought multiple orphan designations and approvals for Humira, the company declined to comment.

Peter Saltonstall, executive director of the National Organization for Rare Disorders, said that Humira is “not a true orphan drug.” But, he said, the company has “the ability to go out and get orphan designation. That’s the way the law reads right now … they can do whatever they want to do.”

It is difficult to say exactly how or if orphan exclusivity affects the price of Humira, which is a complex biologic drug and also has been protected by numerous patents. The drug has long been AbbVie’s top seller, accounting for 63 percent of its revenues, according to its most recent financial filing.

EvaluatePharma notes in its recent report that Humira, as well as a handful of other top drugs, receive less than 25 percent of their sales from orphan uses. Still, if Humira’s orphan uses accounted for just 10 percent of annual sales, the revenue would surpass $1 billion.

By stacking up a series of orphan disease approvals, one seven-year exclusivity period leads into another, maximizing the length of a company’s monopoly. Sigma-Tau Pharmaceuticals, for example, had some form of orphan exclusivity over its metabolic disorder drug for more than 20 years. The drug, Carnitor, received a second orphan approval four months before its first exclusivity was set to expire. And it won its third orphan approval, for an IV formulation of the drug, just one day before its second exclusivity period was set to expire in December of 1999.

“The sequence and timing of regulatory filings for Carnitor reflect the time required to conduct large controlled clinical trials, as well as evolving medical strategies and regulatory pathways pursued by different sponsors over many years,” said GianFranco Fornasini, senior vice president of scientific affairs at Sigma-Tau.

The FDA’s Rao said each new exclusivity period is disease-specific and once any seven-year period runs out, generics can come in. Gleevec, for example, won FDA approval to treat several kinds of rare cancer. All but one of its orphan exclusivity periods had expired by 2015, allowing two generics to enter the marketplace. But Gleevec still has exclusivity until 2020 to treat newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia in patients who are also on chemotherapy.

It’s also true, Rao explained, that some of the drugs that go through the orphan process may not specifically treat a rare disease. For example, a very toxic cancer drug might may not work well in earlier stages because its risks outweigh the benefits. But the company may propose that it will help a smaller group of later-stage cancer patients and win orphan approval just for that group.

Former FDA orphan drug director Haffner said her FDA office worked on rules defining how companies could legitimately pursue approval for a small group of patients with a specific unmet medical need.

“People have played games with the Orphan Drug Act since it was passed,” said Haffner, who first took a job with drugmaker Amgen after leaving the FDA and then became an independent consultant. “It’s the American way, I don’t mean that in a nasty way. But we take advantage of what’s in front of us.”

In reality, Rao said, the regulations did not really change “much of what our practice was.” The agency wanted to address what Rao said were “common misconceptions” and frequently asked questions so officials changed wording in the regulations to better define exactly what could be considered an orphan drug.

Breaking down larger, broader diseases into smaller groups is still allowed under certain conditions and companies can still win multiple orphan approvals for a single drug — even if the total population served rises above the 200,000 mark.

Amgen Inc.’s Repatha won marketing approval and exclusive rights in 2015 for the orphan disease homozygous familial hypercholesterolemia, which affects a population of about 300 people in the U.S. On the very same day, the drug was approved as a mass market drug to treat up to 11 million people with uncontrolled levels of LDL cholesterol, said Amgen spokeswoman Kristen Davis.

Dr. Steven Nissen of the Cleveland Clinic, who ran a broader trial on Repatha, said, “It’s certainly not considered by any of us to be an orphan drug.”

Safeguarding The ‘Prize’

Considering the long history of what’s happened, Tim Coté acknowledges that there are “some loopholes” in the Orphan Drug Act. Perhaps 3 percent or less of approved orphans were not in the “spirit” of the law, he said.

But Coté, rare disease advocates, patients and people in the drug industry expressed fear that changing the Orphan Drug Act or questioning its success would hurt the development of drugs for rare patients.

Former U.S. Rep. Jim Greenwood, R-Pa., now president of the Biotechnology Innovation Organization, an industry trade group, said that concerns about high prices for orphan drugs aren’t justified. The incentives, he said, should not be altered because rare diseases are “tragically killing and brutalizing mostly children.”

Greenwood seemed unaware that dozens of orphan approvals stemmed from the repurposing of mass market drugs, like Humira or Enbrel, another drug developed first for rheumatoid arthritis. Still, he said, “I would argue that the risk of losing incentives in the system far outweighs the benefit of trying to save a few pennies on the health care dollar.”

It’s a sentiment that Coté and other advocates share. While talking about the $311,000 annual price tag for cystic fibrosis drug Kalydeco, Coté said any parent whose child has the disease would be a big fan of the drug.

“The price point is justified because actually it has a dramatic effect on the children. Dead children … people are willing to pay a lot to prevent that,” Coté said. “And that’s a real good thing that we have this drug. OK?”

The first drug to specifically target the underlying biochemical defect of cystic fibrosis, Kalydeco is approved to treat a subset of patients who have specific mutations in their genes. Development of the drug was financed by the Cystic Fibrosis Foundation, which sold its rights to sales royalties from Kalydeco and other cystic fibrosis drugs for $3.3 billion in 2014.

Others, including Henry Waxman, are far more critical and have tried to do something about it over the years. Waxman proposed multiple bills to rein in corporate profits by amending the orphan drug law that he sponsored, but none succeeded.

The FDA has also tried but failed, to keep corporations in check.

In 2012, drugmaker Depomed Inc. filed suit against the FDA for refusing to give its drug Gralise seven years of market exclusivity as a treatment for pain related to shingles.

Rao said the agency wanted to see proof that Gralise was clinically superior to other drugs, noting there “were a bunch of other generics on the market” with different formulations and dosing requirements. Grasile’s active ingredient, gabapentin, is the same one as in Pfizer’s mass market blockbuster Neurontin, which is also approved for treatment of shingles pain.

The FDA approved Gralise but denied seven years of exclusivity.

In response, the drugmaker sued the agency and won its case, arguing that according to the law, they didn’t have to prove their drug was clinically superior to gain the monopoly.

Today, the drug is one of Depomed’s top products with sales of $81 million in 2015. And, in a recent proxy statement, Depomed listed “protecting Gralise exclusivity” as a corporate objective under the category of “enhance and protect future cash flow.” Its orphan exclusivity ends in 2018. Depomed spokesman Christopher Keenan said Gralise wanted patent exclusivity to block competition. But, Keenan said, “Had the patent effort failed on all fronts, the Orphan Drug Designation would have been very important.”

After reviewing KHN’s analysis, Rao said she wants to better understand why drugmakers are applying for multiple approvals and has asked for a case-by-case review of all orphan designations granted in 2010 and 2015. She said the agency lacks the resources to run an analysis of the entire orphan drug database.

“Our goal is to try to get it right,” she said. “There are over 7,000 rare diseases, likely more, the vast majority of which have nothing … I want to ensure that we continue to keep our eye on that prize.”

]]>284943Unexpected Risks Found in Procedure for 3-Person Babyhttps://ww2.kqed.org/futureofyou/2017/01/12/unexpected-risks-found-in-procedure-for-3-person-baby/
Fri, 13 Jan 2017 00:00:57 +0000https://ww2.kqed.org/stateofhealth/?p=283554In September, reproductive endocrinologist John Zhang and his team at the New Hope Fertility Center in New York City captured the world’s attention when they announced the birth of a child to a mother carrying a fatal genetic defect.